EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The Eu­ro­pean Med­i­cines Agency on Fri­day re­ject­ed Ky­owa Kirin’s Parkin­son’s dis­ease drug Nouryant (istrade­fylline), which the US FDA ap­proved in 2019 un­der the brand name Nouri­anz.

EMA said it con­sid­ered that the re­sults of the clin­i­cal stud­ies used to sup­port the ap­pli­ca­tion “were in­con­sis­tent and did not sat­is­fac­to­ri­ly show that Nouryant was ef­fec­tive at re­duc­ing the ‘off’ time. On­ly four out of the eight stud­ies showed a re­duc­tion in ‘off’ time, and the ef­fect did not in­crease with an in­creased dose of Nouryant.”

So-called “off” pe­ri­ods oc­cur when a Parkin­son’s pa­tient has dif­fi­cul­ty mov­ing about and oc­cur when the ef­fect of the last dose of an­oth­er com­mon Parkin­son’s drug, known as lev­odopa, wears off.

“The Agency al­so not­ed that no ef­fect was seen in the two stud­ies that in­clud­ed pa­tients from EU pop­u­la­tions, in­clud­ing the most re­cent study which in­volved pa­tients who were re­ceiv­ing the max­i­mum and op­ti­mal treat­ment for their Parkin­son’s dis­ease,” EMA said.

The FDA in an­nounc­ing its ap­proval not­ed that four, 12-week place­bo-con­trolled clin­i­cal stud­ies that in­clud­ed a to­tal of 1,143 par­tic­i­pants showed Nouri­anz of­fered a sta­tis­ti­cal­ly sig­nif­i­cant de­crease from base­line in dai­ly “off” time com­pared to those on place­bo.

Mean­while, EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use adopt­ed a pos­i­tive opin­ion rec­om­mend­ing mar­ket­ing au­tho­riza­tion for Mod­er­na’s Covid-19 vac­cine, now known as Spike­vax, to in­clude ado­les­cents 12 years of age and old­er. The use of the Spike­vax vac­cine in chil­dren from 12 to 17 years of age will be the same as in peo­ple aged 18 and above, EMA said.

Mod­er­na filed an ap­pli­ca­tion with the FDA for that same younger age group on June 10 but has yet to hear from the agency. States like Rhode Is­land have al­ready signed off on al­low­ing 12 to 17-year-olds to use the vac­cine.

CHMP al­so rec­om­mend­ed for ap­proval Sanofi and Gen­zyme’s new Pompe dis­ease drug, known in Eu­rope as Nexvi­adyme (aval­glu­cosi­dase al­fa). Pompe dis­ease is a rare, in­her­it­ed dis­or­der caused by the buildup of glyco­gen in the body’s cells.

EMA said the drug can help im­prove the res­pi­ra­to­ry func­tion of Pompe dis­ease pa­tients, and the most com­mon side ef­fects in­clude hy­per­sen­si­tiv­i­ty (in­clud­ing ana­phy­lax­is) and in­fu­sion-as­so­ci­at­ed re­ac­tions. In the US, aval­glu­cosi­dase al­fa saw its PDU­FA date pushed back by three months, from May 18 to Aug. 18, ac­cord­ing to a Sanofi in­vestor pre­sen­ta­tion in April.

Though the pro­gram ob­tained pri­or­i­ty re­view from FDA back in No­vem­ber, an­a­lysts ex­pressed skep­ti­cism in Ju­ly 2020 af­ter it failed to prove su­pe­ri­or to the com­pa­nies’ oth­er drug Lu­mizyme in im­prov­ing res­pi­ra­to­ry func­tion in a Phase III tri­al.

CHMP on Fri­day al­so an­nounced that Roche pulled its Tecen­triq ap­pli­ca­tion in triple-neg­a­tive breast can­cer af­ter it not­ed that the EMA said the re­sults from a late-stage tri­al do not fa­vor the drug’s ben­e­fit-risk cal­cu­la­tion in this in­di­ca­tion. In the US, how­ev­er, ODAC in April vot­ed to keep alive the ac­cel­er­at­ed ap­proval for Tecen­triq plus Abrax­ane (nab-pa­cli­tax­el) in mTNBC while ad­di­tion­al con­fir­ma­to­ry tri­als are on­go­ing.

And fi­nal­ly, CHMP al­so con­clud­ed its in­ves­ti­ga­tion in­to blue­bird bio’s Zyn­te­glo, find­ing no ev­i­dence that it caus­es a blood can­cer known as acute myeloid leukemia.

Ear­li­er this month, the drug­mak­er lift­ed a vol­un­tary EU mar­ket­ing hold on Zyn­te­glo for be­ta tha­lassemia, af­ter a pa­tient death in one of the drug­mak­er’s oth­er prod­ucts, Lenti­Glo­bin, halt­ed clin­i­cal stud­ies. Blue­bird de­ter­mined that death was high­ly un­like­ly to have been caused by the de­liv­ery virus used in both Zyn­te­glo and Lenti­Glo­bin.

Blue­bird al­so earned an ap­proval Wednes­day in Eu­rope for its gene ther­a­py Lenti-D for chil­dren with ear­ly cere­bral adrenoleukody­s­tro­phy and an ABCD1 ge­net­ic mu­ta­tion with­out a matched sib­ling donor.

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